Replacing the Enzyme  -L-Iduronidase at Birth Ameliorates Symptoms in the Brain and Periphery of Dogs with Mucopolysaccharidosis Type I

IDS Crossing of the Blood-Brain Barrier Corrects CNS Defects in MPSII Mice

(2009) Vinicia Assunta Polito et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Risk Factor Analysis of Outcomes after Unrelated Cord Blood Transplantation in Patients with Hurler Syndrome

(2009) Jaap Jan Boelens et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation

(2008) L E Polgreen et al.   BONE MARROW TRANSPLANTATION

Tandem Mass Spectrometry for the Direct Assay of Lysosomal Enzymes in Dried Blood Spots: Application to Screening Newborns for Mucopolysaccharidosis I

(2008) S. Blanchard et al.   CLINICAL CHEMISTRY

Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy

(2008) Ramin Sedaghat Herati et al.   MOLECULAR GENETICS AND METABOLISM

Mannose 6-Phosphate Receptor–mediated Transport of Sulfamidase Across the Blood–brain Barrier in the Newborn Mouse

(2008) Akihiko Urayama et al.   MOLECULAR THERAPY

Early Detection of Pompe Disease by Newborn Screening Is Feasible: Results From the Taiwan Screening Program

(2008) Y.-H. Chien et al.   PEDIATRICS